Regulus Therapeutics Inc., a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs (miR), announced that it has expanded its clinical trial collaboration agreement with GSK for the development of RG-101, Regulus’ wholly-owned, GalNAc-conjugated anti-miR that targets miR-122.
In the expanded collaboration, the companies plan to conduct a multi-centered, randomized, dose-ranging Phase II study evaluating the combination of RG-101 and GSK’s long-acting parenteral (LAP) formulation of GSK2878175 as a potential single-visit cure in patients chronically infected with HCV. This study will be conducted outside the United States and is planned to begin in the fourth quarter of 2016. Based on predicted enrollment rates, interim results from this expanded collaboration should be available in the second half of 2017, enabling a potential initiation of a pivotal study in late 2017. As with the initial collaboration, both parties will share equally in the costs associated with the study. Neither Regulus nor GSK has any further obligations or commitments to each other beyond this expanded clinical collaboration agreement.
“We are very pleased to expand our working collaboration with GSK. This is an important next step to advance the scientific understanding of the potential for a combination therapy to achieve a single-visit cure for HCV,” said Paul Grint, M.D., President and CEO of Regulus. “The market research conducted to date indicates that a potential single visit cure would be a highly preferred product profile to existing regimens.”
Zhi Hong, Senior Vice President and Head of the Infectious Diseases Therapy Area, GSK, commented, “We are excited about the potential of this combination to provide people living with HCV a new treatment option that could be delivered in a single visit. Together with Regulus, we are taking another step forward to proving this novel concept.”
Earlier this year, pursuant to the initial GSK-Regulus clinical trial collaboration agreement, Regulus began enrolling patients in an open-label Phase II clinical trial combining RG-101 and GSK2878175 for the treatment of HCV to evaluate the potential to achieve sustained viral responses post treatment with a single subcutaneous administration of 4 mg/kg of RG-101 in combination with daily oral administrations of 20 mg of GSK2878175 for up to 12 weeks in treatment-naïve patients chronically infected with HCV genotypes 1 and 3. Regulus and GSK anticipate reporting interim results from this study by year-end.
The discovery of microRNAs in humans during the last decade is one of the most exciting scientific breakthroughs in recent history. microRNAs are small RNA molecules, typically 20 to 25 nucleotides in length, that do not encode proteins but instead regulate gene expression. More than 800 microRNAs have been identified in the human genome, and over two-thirds of all human genes are believed to be regulated by microRNAs. A single microRNA can regulate entire networks of genes. As such, these molecules are considered master regulators of the human genome. microRNA expression, or function, has been shown to be significantly altered or dysregulated in many disease states, including oncology, fibrosis, metabolic diseases, immune-inflammatory diseases and HCV. Targeting microRNAs with anti-miRs, chemically modified, single-stranded oligonucleotides, offers a unique approach to treating disease by modulating entire biological pathways and may become a new and major class of drugs with broad therapeutic application.
Filed Under: Drug Discovery